- Significant unmet need for a treatment
for cold agglutinin disease, a chronic rare blood disease that can
result in life-threatening events like stroke
- BIVV009 granted breakthrough therapy
designation by the FDA in May 2017
Bioverativ Inc. (NASDAQ: BIVV), a global biopharmaceutical
company dedicated to transforming the lives of people with rare
blood disorders, today announced that the first patient has been
dosed in the Phase 3 clinical program of its investigational
therapy BIVV009 for cold agglutinin disease (CAgD). The Phase 3
program includes two parallel Phase 3 trials, Cardinal and Cadenza,
which are evaluating the efficacy and safety of BIVV009 in adult
patients with primary CAgD, a disease with no approved
therapies.
CAgD is a rare blood disease that results in the premature
destruction of red blood cells (hemolysis) by the body’s immune
system. People with CAgD suffer from anemia, which is often severe,
and can experience profound fatigue, and/or a significantly
increased risk of life-threatening thromboembolic (TE) events such
as stroke, pulmonary embolism, and heart attack. Recently-presented
results from the CAgD Optum study, the largest retrospective study
of cold agglutinin disease, showed that CAgD patients had a 55%
overall increased rate of TE events versus matched controls (31%
vs. 20% p<0.0001), as well as a statistically significant higher
frequency of multiple TE events.1
“I am optimistic that the initiation of the Phase 3 trials of
BIVV009 moves us closer to a new era of treatment for people living
with cold agglutinin disease,” said Catherine Broome, M.D.,
Associate Professor, Georgetown University. “People with cold
agglutinin disease can suffer from a significant disease burden,
and data from the Phase 1 study suggests that BIVV009 may be able
to address the underlying hemolysis, which is at the core of the
disease.”
The Cardinal trial, in which the patient was dosed, is an
open-label, single-arm study that will evaluate the safety and
efficacy of BIVV009 in 20 adult patients with primary CAgD who have
had at least one recent blood transfusion. The Cadenza trial is a
randomized, double-blind, placebo-controlled study that will
evaluate the safety and efficacy of BIVV009 in 40 adult patients
with primary CAgD who have not recently had a blood transfusion.
For more information about the Phase 3 studies, visit
clinicaltrials.gov (study numbers: NCT03347396 and
NCT03347422).
“The start of our Phase 3 trials is an important step toward
bringing the first approved therapy for cold agglutinin disease to
patients who are greatly in need of a safe and effective
treatment,” said Joachim Fruebis, Senior Vice President of
Development at Bioverativ. “Based on results from a Phase 1b trial,
we believe that BIVV009 has the potential to meaningfully improve
the lives of people with CAgD, and we are focused on quickly
advancing the program.”
BIVV009 is a novel monoclonal antibody that has been designed to
block C1s in the classical complement pathway and directly target
the root cause of cold agglutinin disease. It is believed that this
approach may prevent the complement system, which is a part of the
immune system, from mistakenly destroying a person’s red blood
cells, and potentially halt the CAgD disease process.
BIVV009 was awarded Breakthrough Therapy Designation by the U.S.
Food and Drug Administration (FDA) and Orphan Drug Designation by
both the FDA and European Medicines Agency. These designations
acknowledge the potential of novel drugs and biologics, and are
intended to expedite the development and regulatory review of such
therapies.
About the BIVV009 Phase 1b StudyThe Phase 1b study
assessed the long-term efficacy, safety, and PK/PD profile of
BIVV009 in six severely anemic primary CAgD patients. Primary and
secondary outcome measures were achieved in the CAgD patients in
the study. Hemoglobin levels increased in all six patients (median
>4g/dl), eliminating the need for transfusions while on
treatment. Maintenance therapy through a Named Patient Program has
demonstrated a sustained response for more than 18 months,
including control of hemolysis. Safety data through December 21,
2016, demonstrated that BIVV009 was generally well tolerated. Five
of six patients (83.3%) in the primary CAgD group experienced at
least one adverse event (AE); no AE was reported by more than one
patient. One unrelated, serious AE occurred in a patient with CAgD
who was hospitalized for a pre-existing condition. There were no
serious AEs assessed as related to BIVV009 by the investigator.
About Cold Agglutinin Disease (CAgD)CAgD is a severe,
chronic rare blood disease in which a part of the body’s immune
system called the complement system mistakenly attacks a person’s
own red blood cells. People with CAgD suffer from chronic anemia,
severe fatigue, and an increased risk of life-threatening events
such as stroke. There are no approved therapies for CAgD, which
occurs in approximately 16 people per million,2 including an
estimated 10,000 people in the United States and Europe. Current
treatment options are aimed at normalizing hemoglobin levels
through blood transfusions, immunomodulating therapy not approved
for CAgD, or chemotherapy using cytotoxic agents. These options
have significant toxicity risks and are often nondurable, leaving
patients dependent on frequent transfusions, which can lead to
chronic iron overload. Learn more about CAgD.
About BIVV009BIVV009 is a first-in-class, humanized,
monoclonal antibody that has been specifically designed to target
C1s, a serine protease within the C1-complex, that is the first
step in activating the classical complement pathway of the immune
system. The classical complement pathway is the central mechanism
responsible for the development of hemolytic anemia in cold
agglutinin disease and blocking it may potentially halt the CAgD
disease process. With a novel mechanism of action and high target
specificity, BIVV009 is designed to selectively inhibit disease
processes upstream in the classical complement pathway while
leaving intact the alternative and lectin complement pathways and
their immune surveillance functions.
About BioverativBioverativ is a global biopharmaceutical
company dedicated to transforming the lives of people with
hemophilia and other rare blood disorders through world-class
research, development, and commercialization of innovative
therapies. Launched in 2017 following separation from Biogen Inc.,
Bioverativ builds upon a strong heritage of scientific innovation
and is committed to actively working with the blood disorders
community. The company’s mission is to create progress for patients
where they need it most, and its hemophilia therapies when launched
represented the first major advancements in hemophilia treatment in
more than two decades. For more information, visit
www.bioverativ.com or follow @bioverativ on Twitter.
Bioverativ Safe HarborThis press release contains
forward-looking statements, including statements about the
potential benefits and improvements in the lives of people with
CAgD who are treated with BIVV009. These forward-looking statements
may be accompanied by such words as “anticipate,” “believe,”
“could,” “estimate,” “expect,” “forecast,” “intend,” “may,” “plan,”
“potential,” “project,” “target,” “will” and other words and terms
of similar meaning. You should not place undue reliance on these
statements. Drug development and commercialization involve a high
degree of risk, and only a small number of research and development
programs result in commercialization of a product. Results in early
stage clinical trials may not be indicative of full results or
results from later stage or larger scale clinical trials and do not
ensure regulatory approval. Factors which could cause actual
results to differ materially from Bioverativ's current expectations
include: uncertainties relating to the initiation, enrollment and
completion of stages of clinical trials; reliance on third parties
for aspects of clinical trials; unexpected concerns may arise from
data, analysis or results obtained during clinical trials or post
hoc analysis of studies; regulatory authorities may require
additional information or further studies, or may fail or refuse to
approve or may delay approval of product candidates; or Bioverativ
may encounter other unexpected hurdles; and other risks and
uncertainties associated with Bioverativ’s drug development and
commercialization activities described in the Risk Factors section
of Bioverativ’s filings with the Securities and Exchange
Commission. These statements are based on Bioverativ’s current
beliefs and expectations and speak only as of the date of this
press release. Bioverativ does not undertake any obligation to
publicly update any forward-looking statements.
References1. Broome, C., et al. Incidence of Thromboembolic
Events Is Increased in a Retrospective Analysis of a Large Cold
Agglutinin Disease (CAD) Cohort. 59th Annual Meeting of the
American Society of Hematology; December 9-12, 2017; Atlanta.
2. Mullins M, et al. Cold agglutinin disease burden: a
longitudinal analysis of anemia, medications, transfusions, and
health care utilization. Blood Advances. 2017;1(13):839-848
View source
version on businesswire.com: http://www.businesswire.com/news/home/20180306005377/en/
Bioverativ Inc.Media Contact:Tracy Vineis, +1
781-663-4376media@bioverativ.comorInvestor Relations Contact:Samuel
Chase, +1 781-663-4360IR@bioverativ.com
Bioverativ Inc. (NASDAQ:BIVV)
Historical Stock Chart
From May 2024 to Jun 2024
Bioverativ Inc. (NASDAQ:BIVV)
Historical Stock Chart
From Jun 2023 to Jun 2024